Using tiny materials for gene therapy and genome editing: challenges and new approaches

Nanomaterials provide structural and functional advantages for delivering nucleic acids and CRISPR/Cas systems across biological barriers.

• The behavior of nanomaterials within living systems can be unpredictable, leading to potential off-target editing and immune responses.
• Designing nanocarriers requires a balance between efficiency and safety, integrating precision in their physical properties with adaptability to biological environments.
• Ionizable lipid nanoparticles have shown that adjusting charge, surface chemistry, and degradation rates can improve delivery performance, though issues with reproducibility and scalability remain.
• Polymeric and exosome-inspired systems offer the potential for targeted reuse but require more research into their long-term biocompatibility and acceptance by regulatory bodies.
• Advancements in genome engineering using nanomaterials will depend on optimizing delivery methods, predicting nano-bio interactions, and establishing standardized evaluation processes.

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